I copied this from a post by Dxavier on the Nktr board:
LINCOLNSHIRE, Ill.--(BUSINESS WIRE)--BioSante Pharmaceuticals, Inc. (NASDAQ: BPAX - News) today announced positive GVAX AML Vaccine results and BioSante’s receipt of Orphan Drug designation from the FDA’s Office of Orphan Products Development for GVAX AML Vaccine in the treatment of acute myeloid leukemia (AML). This Orphan Drug designation is the second GVAX cancer vaccine from BioSante to secure FDA orphan drug designation.
In a paper published in the peer-reviewed journal Blood, clinical investigators, led by investigators at Johns Hopkins University, reported on the results of a Phase II study of GVAX AML (immunotherapy) accompanied by immunotherapy-primed lymphocytes after autologous stem cell transplantation in hematologic malignancies. Fifty-four subjects were enrolled, with 28 (52%) receiving a pre-transplantation GVAX AML dose. A total of 46 (85%) subjects achieved complete remission during the treatment period. For all patients who achieved complete remission, the 3-year relapse-free survival (RFS) rate was 47.4% compared to 61.8% in the GVAX-treated group. While the overall survival rate in all subjects was 57.4%, it was 73.4% in the GVAX-treated group. The authors of the paper conclude, “…immunotherapy in combination with primed lymphocytes and autologous stem cell transplantation shows encouraging signals of potential activity in acute myeloid leukemia.”
“The GVAX AML results reported in the medical journal Blood are very exiting. Those patients treated with GVAX had both a relapse-free survival and overall survival rate improvement of approximately 30% compared with the non-GVAX-treated subjects. The GVAX AML results reported today are in addition to the results recently reported for GVAX CML (Chronic Myeloid Leukemia),” said Stephen M. Simes, BioSante’s president & CEO. “These encouraging efficacy data along with our second GVAX orphan drug designation should attract increased attention to our portfolio of GVAX cancer vaccines.” The paper on GVAX AML results appeared in the peer-reviewed medical journal of the American Society of Hematology, Blood (2009; 114:1736-1745).
The Orphan Drug designation of GVAX Acute Myeloid Leukemia Vaccine for the treatment of acute myeloid leukemia is BioSante’s second GVAX regulatory submission and response from the FDA since acquiring this portfolio of cancer vaccines last October. On March 15, BioSante reported receiving orphan drug designation for its GVAX Pancreas Vaccine to treat pancreatic cancer.
“It is our intention to find ways to continue the development of our GVAX cancer vaccines using the benefits conferred by gaining Orphan Drug designation. Further, these orphan drug designations alert the FDA that we are dedicated to bringing better cancer therapy to patients in need,” Simes continued.
As you perhaps already know, this is not the diagnosis that Ceplene is working against. See for example the list of potential patients in the US in the last webcast. Look at the last slides where the transplanted patient is moved away as potential patients. (They constitutes some 20% of all patients.)
("The authors of the paper conclude, “…immunotherapy in combination with primed lymphocytes and autologous stem cell transplantation shows encouraging signals of potential activity in acute myeloid leukemia.”")
The more patient that recieve first remission, the more patient have a potential demand for a cure versus relaps from that paus from the cancer. That use to be the most common answer to new treatment for AML.
When do the new 12 year legislation start to be the prevailing law? Direct after the signing? Anyone, please.
Yes, you are right and I did know that. This treatment looks like an improved way to reach full remission but relapses occur eventually, as with other treatments. Those patients in full remission who want to delay remission could still be served by Ceplene. A question is, how many patients will relapse? At the moment, it is most. If this treatment reduced eventual relapse per se, it could reduce the perceived need for ceplene. But this seems unlikely.